Tumorablative conditioning regimen for haploidentical stem cell transplantation in 102 children with hematologic malignancies: a single-center experience

Abstract

Haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) is widely carried out in China, and transplantation related complications decreased gradually with the transplant technology improving, and the overall survival(OS) increased year by year. However, relapse after transplantation is still one of the main causes of death in patients with hematological malignancy. In order to reduce the recurrence after HSCT, we set a tumorablative conditioning regimen (TAC ) regimen; the aim is as much as possible to eliminate the malignant clone to reduce the recurrence without increasing the conditioning toxicity. We retrospectively analyzed 102 cases of haplo-HSCT in our hospital from 2012 to 2017. Ninety-eight out of the 99 (99.0%) patients achieved primary engraftment. The 2-year OS and disease free survival (DFS) are 81.4% (83/102) and 77.45% (79/102). The cumulative incidence of leukemia relapse is 16.2% (16/99), Twenty-nine patients developed II-IV acute graft-versus-host disease (aGVHD) (29%) within 100 days and only nine patients have grade III-IV aGVHD (9%) in measurable 99 patients. The conditioning regimen was relatively well tolerated with limited regimen-related toxicity. The preliminary results show that TAC is safe and effective in haplo-HSCT of children with hematologic malignancies. This study will provide a clinical basis for the individualized conditioning regimen.

Juvenile myelomonocytic leukemia in the molecular era: a clinician’s guide to diagnosis, risk stratification, and treatment

“…The use of haploidentical donors for this condition is largely anecdotal. 34,69 Therefore, UCB and haploidentical transplants should only be attempted at centers with expertise in those approaches. Although JMML-specific data are lacking, by analogy to AML, if multiple suitable donors are available, centers may consider killer immunoglobin-like receptors testing to potentially optimize natural killer cell-mediated GVL effects.…”